Gene Therapy: SCHN leaders in Advanced Therapeutics

We are Australia's leaders in Paediatric Gene Therapy. 

Sydney Children’s Hospitals Network (SCHN) is the largest network of hospitals and services for children in Australia. Along with our Luminesce Alliance (LA) partners, we are national leaders in diagnostic, data and disease modelling research, with the knowledge and expertise that will be required to develop, evaluate and deliver novel disruptive therapies for rare disorders in children.

Get in touch with us today for advice and assistance on paediatric gene therapy clinical trials.

Contact us

Gene Therapy Leads

• Prof Ian Alexander
  Head of the Gene Therapy Research Unit, initiative of Children’s Medical Research Institute (CMRI) and The Children’s Hospital at Westmead
• Dr Leszek Lisowski
  Head of Translational Vectorology Unit and Manager of Vector and Genome Engineering Facility at CMRI

Clinical researchers (non-cancer; rare diseases)

• A/Prof Michelle Farrar
  Associate Professor in Paediatric Neurology at UNSW Sydney and specialist child neurologist at Sydney Children’s Hospital, Randwick
• Prof Robyn Jamieson
  Head of the Eye Genetics Research Program, a partnership between The Children’s Hospital at Westmead, CMRI, Save Sight Institute, Sydney Eye Hospital and the University of Sydney and the Sydney Genome Diagnostics Laboratory
• Prof Stephen Alexander
  Chronic Kidney Disease Laboratory Research Group Leader and Professor in Paediatrics & Child Health at the Children’s Hospital at Westmead
• Dr Wendy Gold
  Molecular Neurobiology Research Lab Group Leader and Senior Research Fellow, The Children’s Hospital at Westmead

Clinical researchers (cancer)

• Prof Tracey O’Brien
  Director of the Kids Cancer Centre and Head of the Transplant and Cellular Therapies Program at Sydney Children's Hospital, Randwick
• Dr Richard Mitchell
  Deputy Director of the Blood and Marrow Transplantation Program at the Kids Cancer Centre and Paediatric Oncologist at Sydney Children's Hospital, Randwick
• A/Prof David Ziegler
  Deputy Director of the Kids Cancer Alliance, Group Leader at the Children’s Cancer Institute and Senior Staff Specialist in the Kids Cancer Centre at Sydney Children’s Hospital
• Dr Luciano Dalla-Pozza
  Head of the Cancer Centre for Children and Paediatric Oncologist t at The Children’s Hospital at Westmead
• Prof Peter Shaw
  Head of Bone Marrow Transplant Services and Paediatric Oncologist at The Children's Hospital at Westmead 
• Dr Geoff McCowage
  Cancer Gene Therapy Group Leader and Paediatric Oncologist at The Children’s Hospital at Westmead

Clinical trial medical leads

• Prof Craig Munns
  Endocrinologist and Advanced Therapeutics Lead at The Children’s Hospital at Westmead
• Dr Laura Fawcett
  Respiratory Paediatrician and Advanced Therapeutics Lead at Sydney Children’s Hospital, Randwick

Pharmacy lead (gene therapy)

• Peter Barclay
  Pharmacy Director at The Children's Hospitals at Westmead

Early Phase Clinical Trials Contact

• Amy Boland
  Early Phase Clinical Trials Program Manager

Early Phase Human Research Ethics Committee

• Asra Gholami
  Executive Officer, SCHN HREC

Background on gene therapies in SCHN

Rare genetic diseases, although individually uncommon, collectively affect approximately 5% of the population. For the majority there is no cure but for the first time, gene therapies are transforming the lives of children with rare genetic and acquired disorders by offering a cure.

What is Gene Therapy?

Gene therapy is a process that introduces genetic material into cells to replace or compensate for abnormal genes and/or to make a beneficial protein. More specifically, modified viruses such as adeno-associated virus (AAV) and lenti virus are used as vectors to transport the gene replacement DNA to the target tissue and then the protein production.

Successful treatment through gene therapies

Gene therapies are emerging as a major game changer in medicine, proven to be effective therapies for previously untreatable diseases, watch our full webinar here.

Our successful breakthrough study of Australia’s first Spinal Muscular Atrophy (SMA) trial by gene therapy, screened over 200,000 babies across New South Wales. The study showed the importance of early genetic identification for this devastating disease, but also the effectiveness of personalised medicine, such as gene therapy as a treatment. 

The transformative capabilities of gene therapies are evident, with The NSW Government investing $25 million to build an advanced viral vector facility at our Westmead Health Precinct, this will give patients with genetic diseases, cancers and viral infections across Australia faster access to ground breaking trial therapies.

Additionally, nine gene and cell therapy projects across the Westmead Health Precinct (SCHN, CMRI, The Westmead Institute for Medical Research (WIMR)) have been awarded funding to offer PhD scholarships for graduates to undertake a commercialization training program to improve their understanding of the translational pathway of these therapies. Luminesce Alliance has also announced their Paediatric Precision Medicine Seed Funding for SCHN leading researchers to bring research from the lab to the clinics, giving patients faster access to these ground breaking therapies.

Leading expertise at Kids Research, SCHN

Our expertise at Kids Research, SCHN and our unique position with partners across Australia, along with existing clinical trial experiences are enabling us to be leaders in the delivery of gene therapies for children nationally.